With the submission of regulatory marketing applications for its once-daily vanzacaftor triple, also known as the vanza triple program (vanzacaftor/tezacaftor/deutivacaftor) with the European Medicines Agency (EMA) and the US FDA, Vertex Pharmaceuticals is expected to hold its lead in the development of therapies for the treatment of cystic fibrosis (CF). Approval and success of vanza triple could further solidify Vertex’s dominance in CF treatment, says GlobalData
Vertex’s portfolio of cystic fibrosis transmembrane conductance regulator (CFTR) modulators, including Kalydeco (ivacaftor), Orkambi (lumacaftor/ivacaftor), Symdeko/Symkevi (tezacaftor/ivacaftor and ivacaftor), and Trikafta/Kaftrio (elexacaftor/tezacaftor/ivacaftor and ivacaftor), has transformed the CF market by targeting the root cause of the disease.
Sravani Meka, Senior Immunology Analyst at GlobalData, comments, “Among the therapies, Trikafta/Kaftrio stands out as the most effective treatment, addressing a broader range of mutation profiles (with at least one F508del mutation or at least one other mutation in the CF gene) compared to earlier CF therapies. The potential approval of vanza triple could extend Vertex’s leadership in the CF market, especially with Trikafta’s patent expiration not anticipated until 2037. Filed in February 2022, the patent for vanza triple positions Vertex to maintain its market dominance with a new standard of care for CF patients.”
The submission of regulatory marketing applications follows the successful Phase III trials (SKYLINE 102, SKYLINE 103, and RIDGELINE 105), where vanza triple met its primary endpoints, demonstrating noninferiority to Trikafta in improving lung function and superiority in reducing sweat chloride (SwCl) levels. While the direct correlation between SwCl levels and lung function improvement remains to be fully understood, vanza triple presents a promising alternative for patients who experience side effects with Trikafta, such as rash, liver enzyme dysregulation, and CNS issues or prefer a once-daily dosing regimen.
Market projections by GlobalData estimate Trikafta sales to reach $10.4 billion by 2030, with vanza triple expected to generate $5.5 billion in the same period, surpassing other Vertex CFTR modulators. These projections are driven by the high cost of treatment, with Trikafta averaging $948 per treatment day, translating to approximately $346,000 annually.
Despite its success, Vertex faces pricing pressures and challenges over drug affordability and access. In April 2024, Vertex announced that it reached an agreement in South Africa to provide access to select patients via a private health plan following criticism over alleged patent abuse over the lack of Trikafta generics in South Africa, despite holding patents since 2019.
More recently, the company appointed Dr Jennifer Schneider, current Co-founder & CEO of Homeward Health, a company focused on the delivery of health and care in partnership with communities everywhere, particularly in rural America, to its board of directors. By leveraging her vast expertise in health systems, policy, and reimbursement, Vertex may look to navigate these challenges.
Meka concludes, “CF treatment has transformed due to advances in disease understanding and targeted therapies. Vertex, with its CFTR modulators, has revolutionised treatment, notably with Trikafta/Kaftrio. The company’s regulatory submission for the once-daily vanza triple highlights its commitment to innovation. Although vanza triple shows promise in clinical trials, its market impact is uncertain amid pricing pressures and advocacy group challenges.
“Vertex’s strategic moves, including negotiations with South Africa over the access of Trikafta and additions to its senior leadership, reflect its proactive stance in maintaining market leadership.”
Edits made by EP News Bureau